Gene Therapy Using AAV8hAATAVL to Treat Alpha 1-Antitrypsin Deficiency in Adults

What this Trial Is About

Researchers are evaluating a gene therapy called AAV8hAATAVL to treat alpha 1-antitrypsin AAT deficiency. This condition affects the lungs due to a deficiency of the AAT protein. The study aims to deliver a single intravenous dose of this gene therapy, which codes for an oxidation-resistant form of the AAT protein, to see if it can safely and effectively protect the lungs over time. Participants will receive different doses of the gene therapy, with groups receiving varying amounts of vector genome copies per kilogram of body weight. The study is non-randomized and open-label, with no placebo group. The treatment is given as a one-time intravenous infusion, and researchers will observe the participants for safety, toxicity, and dosage tolerance over approximately two years. During the study, participants will undergo various assessments including blood tests to measure AAT levels in serum and lung fluid at multiple time points up to five years. Safety will be monitored by tracking serious adverse events and dose-limiting toxicities. Lung function will be evaluated by imaging and clinical tests. The total participation may last several years to gather long-term data on the gene therapys effects and safety.

Gene Therapy for Alpha 1- Antitrypsin Deficiency